mRNA technology could be possible treatment for rare diseases
By exploiting the technology used in Covid-19 vaccines, a team led by UCL, King’s College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology’s potential therapeutic use in people.
The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model of the disease.
Argininosuccinic aciduria is an inherited metabolic disorder that affects how the body ...












